Content » Vol 84, Issue 5

Clinical Report

Enzyme Replacement Therapy in Severe Fabry Disease with Renal Failure: A 1-year Follow-up

Dionysios Tsambaos , Elisabeth Chroni , Antonis S. Manolis , Alexandra Monastirli , Efi Pasmatzi , Theophilos Sakkis , Periklis Davlouros , Dimitrios Goumenos , Aggeliki Katrivanou , Sophia Georgiou
DOI: 10.1080/00015550410030682


Wepresent here the course of clinical response of a 53-year-oldhaemodialysed Fabry patient who received recombinant human α-galactosidase A at a dose of 1 mg/kg every other week over a period of 1 year. The therapy was well tolerated by the patient, who revealedan impressive favourable cutaneous, gastrointestinal, neurological andpsychiatric response and a dramatic improvement in his quality of life,but no improvement in cardiac and renal function.


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